Biogen has revealed encouraging phase 2 results with its anti-CD38 antibody felzartamab in rare kidney disease IgA nephropathy (IgAN), which it hopes could join a series of first-in-class t
Novartis is cueing up a new indication in the US for its oral therapy for complement-mediated diseases – Fabhalta – after reporting new data with the drug in ultra-rare kidney disease C3 gl
AstraZeneca and Ionis are set fair for EU approval of Wainzua, their treatment for polyneuropathy associated with transthyretin-mediated amyloidosis (ATTR), after the EMA's human medicines
Neuroscience specialist Lundbeck has agreed to buy Longboard Pharmaceuticals for $2.6 billion, expanding its pipeline with an epilepsy drug tipped as a future blockbuster.
Zealand Pharma has suffered another setback in its bid to bring glucagon receptor agonist dasiglucagon for ultra-rare disease congenital hyperinsulinism (CHI) to the US market.
In the space of four days, the number of FDA-approved therapies for ultra-rare genetic disorder Niemann-Pick disease has gone from zero to two, transforming the prospects for patients.
It has been five years since Lexicon Pharma's type 1 diabetes therapy, Zynquista, was rejected by the FDA, and it may struggle to have a better outcome at its second attempt.